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Table 3 Overview of included OMPs

From: Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

Drug Generic name Year of authorization Type of authorization Disease Specification of indication
Gliveca imatinib 2002
Exceptional 1. GIST
1. Unresectable or metastatic GISTs.
2. Unresectable or metastatic DFSP, and adults who are not eligible for surgery after disease recurrence.
Lysodrena mitotane 2004 Full Adrenocortical carcinoma Unresectable, metastatic or relapsed adrenocortical carcinoma.
Sutenta sunitinib 2006
Conditional 1. GIST
2. RCC
1. Unresectable or metastatic GISTs. Used after treatment with imatinib has failed.
2. Metastatic RCC. Patients who failed to respond to or relapsed after IFN-a or IL-2 based therapy.
Nexavara sorafenib 2006
Full 1. RCC
2. HCC
3. Differentiated thyroid carcinoma
1. Advanced RCC. Patients who have failed to prior IFN-a or IL-2 based therapy.
2. Not specified.
3. Locally advanced or metastatic differentiated thyroid carcinoma refractory to RAI.
Torisel temsirolimus 2007 Full RCC Advanced RCC. Patients who have not previously received systemic therapy and who have at least 3 of 6 prognostic risk factors.
Yondelis trabectedine 2007
Exceptional 1. STS
2. Ovarian cancer
1. Advanced STS, patients in which anthracyclines and ifosfamide failed, or who are unsuited to receive these agents.
2. Relapsed ovarian cancer, sensitive to platinum-based chemotherapy. Used in combination with pegylated liposomal doxorubicin.
Mepact mifamurtide 2009 Full Osteosarcoma High-grade resectable non-metastatic osteosarcoma after macroscopically complete surgical resection. Combined with post-operative multi-agent chemotherapy.
Afinitora everolimus 2009 Full RCC Advanced RCC when relapsed after treatment with a VEGF-targeted medicine.
Cometriq cabozantinib 2014 Conditional Medullary thyroid cancer Progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma.
Lynparza olaparib 2014 Full Ovarian cancer Maintenance therapy of adult patients with platinum-sensitive relapsed BRCA-mutated ovarian cancer who are in response to platinum-based chemotherapy.
Cyramzaa ramucirumab 2014 Full Gastric cancer Advanced gastric cancer or gastro-oesophageal junction adenocarcinoma with disease progression after prior platinum and fluoropyrimidine chemotherapy. Combined with paclitaxel.
Unituxinb dinutuximab 2015 Full Neuroblastoma Patients aged 11 months to 17 years with high-risk neuroblastoma, following myeloablative therapy and ASCT, in combination with GM-CSF, IL-2 and isotretinoin.
Lenvima lenvatinib 2015 Full Differentiated thyroid carcinoma Progressive, locally advanced or metastatic differentiated (papillary/follicular/Hürthle cell) thyroid carcinoma, refractory to RAI.
Lartruvo olaratumab 2016 Conditional STS Adult patients with advanced STS, not amenable to curative treatment with surgery or radiotherapy and not previously treated with doxorubicin. Used in combination with doxorubicin.
Onivyde nanoliposomal irinotecan 2016 Full Pancreatic cancer Metastatic adenocarcinoma of the pancreas, in combination with 5-FU and LV, in adult patients who have progressed following gemcitabine based therapy.
  1. 5-FU 5 fluorouracil, ASCT autologous stem cell transplant, BRCA breast cancer, DB double-blind, DFSP dermatofibrosarcoma protuberans, EFS event-free survival, GIST gastrointestinal stromal tumor, GM-CSF granulocyte-macrophage colony-stimulating factor, HCC hepatocellular carcinoma, Her2Neu Human Epidermal growth factor Receptor 2, HR hormone receptor, IFN-a interferon-alpha, IL-2 interleukin-2, LV leucovorin, OMP orphan medicinal product, OS overall survival, ORR objective response rate, Pbo placebo, PFS progression-free survival, pNET pancreatic neuroendocrine tumor, RAI radioactive iodine, RCC renal cell carcinoma, RCT randomized controlled trial, RFS recurrence-free survival, SAT single-arm trial, STS soft tissue sarcoma, TTP time to progression, VEGF vascular endothelial growth factor
  2. aNo longer an orphan drug. Withdrawn from the Community register of OMPs upon request of the marketing authorisation holder or after 10 years of market exclusivity.
  3. bWithdrawn from use in the European Union at the request of the marketing authorisation holder due to short- and intermediate- term inability to supply Unituxin in sufficient quantities for meeting current global demands
  4. All information in this table was retrieved from